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Published online February 4, 2008
PEDIATRICS (doi:10.1542/peds.2007-0665)
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EXPERIENCE & REASON

Successful Management of Difficult Infusion-Associated Reactions in a Young Patient With Mucopolysaccharidosis Type VI Receiving Recombinant Human Arylsulfatase B (Galsulfase [Naglazyme])

Katherine H. Kim, MSa,b, Celeste Decker, MDc, Barbara K. Burton, MDa,b

a Division of Genetics, Birth Defects, and Metabolism, Children's Memorial Hospital, Chicago, Illinois
b Department of Pediatrics, Feinberg School of Medicine, Northwestern University, Chicago, Illinois
c BioMarin Pharmaceutical Inc, Novato, California

ABSTRACT

Our patient with mucopolysaccharidosis type VI received enzyme replacement therapy with recombinant human arylsulfatase B (galsulfase [Naglazyme, BioMarin Pharmaceutical Inc, Novato, CA]) shortly after approval by the US Food and Drug Administration. After 1 month of weekly infusions, the patient developed significant infusion-associated reactions and could not tolerate therapy at the recommended infusion rate. We were able to continue treatment successfully by the addition of steroids to the premedication regimen and by significantly reducing the rate of drug infusion. Over the next several months, the patient's infusion rate was slowly increased and the premedications were weaned. We demonstrate that by significantly reducing the rate of infusions and adjusting the premedication regimen, galsulfase infusions can continue with no additional observance of infusion-associated reactions.

Key Words: MPS VI • management • enzyme replacement therapy • recombinant human arylsulfatase B • galsulfase • Naglazyme

Abbreviations: MPS VI, mucopolysaccharidosis type VI • ASB, arylsulfatase B • GAG, glycosaminoglycan • ERT, enzyme replacement therapy • IAR, infusion-associated reactions

Accepted Jul 27, 2007.


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